Verona Pharma recently announced the company’s accomplishments in the last six months, including an update to the company’s clinical trial in cystic fibrosis (CF) and positive interim results for its investigational therapy RPL554. RPL554 is an inhaled dual inhibitor of two enzymes, phosphodiesterase 3 and 4, and was previously shown to…
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The U.S. Food and Drug Administration (FDA) is advising patients and healthcare professionals to not use any liquid products made by PharmaTech, LLC due to possible contamination with Burkholderia cepacia bacteria, which may cause serious infections in patients with chronic lung diseases, including cystic fibrosis (CF). The regulatory agency previously had…
Concert Pharmaceuticals’ sale of its cystic fibrosis therapy CTP-656 to the larger Vertex Pharmaceuticals will lead to cystic fibrosis patients obtaining faster access to the therapy, Concert CEO Roger Tung predicted. He also said that the $160 million that Concert obtained from the sale will allow the company to advance its hair-loss therapy alopecia areata CTP-543 into…
AIT Therapeutics has administered its first doses of nitric oxide in a Phase 2 clinical trial of the compound’s ability to kill the drug-resistant bacteria often found in cystic fibrosis patients. The study’s overarching goal is to see whether AIT’s nitric oxide formulation can eliminate nontuberculous mycobacteria, or NTM, in patients…
Sweat from infants with cystic fibrosis contains substances that could be used as biomarkers of the disease and even identify new disease mechanisms, a study reports. The research, published in the journal ACS Central Science, was titled “The Sweat Metabolome of Screen-Positive Cystic Fibrosis Infants: Revealing Mechanisms beyond Impaired Chloride Transport.” Scientists…
A new method of isolating lung stem cells could help speed the development of stem-cell based therapies for lung diseases, including cystic fibrosis, according to a University of North Carolina study. That method is extracting them with a tube from the mouth to the lung rather than surgery. The team…
In a groundbreaking study, scientists reported editing and correcting, for the first time, a defective gene in human embryos. Additionally, this gene-editing technique appeared to be safely performed without introducing additional harmful genetic alterations. These results represent a key achievement in human genetic engineering and highlight a potential way…
Two of the three therapies that Proteostasis Therapeutics has developed to correct the underlying problem in cystic fibrosis are going through clinical trials and a third is heading for trials, the company reports. That underlying problem is a mutation in a gene known as the cystic fibrosis transmembrane conductance regulator, or CFTR. It produces…
Treatment with Alcresta Therapeutics’ product Relizorb effectively increased fat absorption in patients with cystic fibrosis (CF) receiving enteral tube feeding who have trouble breaking down and absorbing these molecules. This resulted in a reduction of the frequency and severity of gastrointestinal symptoms. These findings were published recently in the…
The U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) to treat more than 600 of the estimated 30,000 Americans with cystic fibrosis (CF), Vertex Pharmaceuticals announced. The oral medicine may now be prescribed to CF patients ages 2 and older who have one of five residual…
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